Late-Stage Clinical Trials for AstraZeneca's Amyloidosis Drug Faces Setbacks
In a significant development for the treatment of amyloidosis, a rare and potentially life-threatening disease that damages organs, particularly the heart and kidneys, AstraZeneca's rare disease division, Alexion, is testing Anselamimab, an antibody, in two global Phase III trials called Cardiac Amyloid Reaching for Extended Survival (CARES).
The drug, which targets and aims to eliminate amyloid deposits, has received fast track and orphan drug designation from the FDA for treating amyloidosis. Anselamimab has also received orphan drug tracks in the European Union and Japan.
The trials, which enrolled 406 patients with light chain amyloidosis, did not meet their primary endpoint of improving survival or reducing hospitalizations across the board. However, a prespecified patient subgroup, consisting of those with Mayo stages IIIa and IIIb light chain (AL) amyloidosis, demonstrated a clinically meaningful improvement in time to all-cause mortality (ACM) and frequency of cardiovascular hospitalizations (CVH) compared to placebo.
The company did not initially specify what this patient subgroup was, but in a recent update, AstraZeneca reported a clinically meaningful improvement for this group. The company is currently performing a fuller data analysis of the Anselamimab trials and will present the results at an upcoming medical meeting.
AstraZeneca acquired Anselamimab in 2021 when it purchased Caelum Biosciences. Meanwhile, the company also acquired Baxdrostat through its 2023 purchase of CinCor Pharma. Baxdrostat, a treatment for uncontrolled or treatment-resistant hypertension, has shown promise in a Phase III trial, pushing blood pressure down in patients enrolled.
In July, Alexion gained access to a suite of new AAV vectors from JCR Pharmaceuticals to support its gene therapy program. The drug, Anselamimab, was well tolerated, with most adverse events balanced between the treatment and the placebo arms.
This development suggests that Anselamimab may provide a meaningful benefit in more advanced cases of AL amyloidosis, offering hope for patients battling this debilitating disease.
Science in the field of medical-conditions like amyloidosis is making strides with drug development. AstraZeneca's Anselamimab, an antibody under investigation, has shown promising results in a subgroup of patients with Mayo stages IIIa and IIIb light chain (AL) amyloidosis, indicating its potential for health-and-wellness improvements in advanced cases of this disease.
